Agenus Reports First Quarter 2018 Financial Results and Provides Corporate Update


On May 7, 2018 Agenus Inc. (NASDAQ: AGEN), an immuno-oncology (I-O) company with a pipeline of immune checkpoint antibodies, cancer vaccines and adoptive cell therapies1, provided a corporate update and reported financial results for the first quarter ending March 31, 2018 (Press release, Agenus, MAY 7, 2018, View Source [SID1234526159]).

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"Innovation and speed are the basis of our I-O business model with 5 INDs filed over the past 18 months, 6 INDs on track for this year and 2 additional INDs planned in the 1H of next year. We have treated over 100 patients with our CTLA-4 (AGEN1884) and/or PD-1 (AGEN2034) antibodies with clinical responses in some of the patients with advanced cancers," said Garo H. Armen, Ph.D., Chairman and CEO of Agenus. " We have also made progress with our commercial readiness with commercial grade antibodies already produced; our partnering discussions are maturing, and we are committed to bring these discussions to closure."

Milestones Achieved and Upcoming

Clinical update:
To date, we have:
Presented AGEN1884 (CTLA-4) & AGEN2034(PD-1) pharmacodynamic activity at AACR (Free AACR Whitepaper)2018;
Presented preclinical data on TIM-3 (INCAGN02390) and LAG-3 (INCAGN02385); Clinical trials planned for 2018;
Completed dose escalation of AGEN1884 & AGEN2034 combination;
Launched combination trials with AGEN1884 & AGEN2034, including trials in 2L cervical cancer.
In the coming year, we expect to:
Present efficacy data on AGEN1884 and AGEN2034
>100 patients treated; clinical activity observed;
Interim data review suggests patients with advanced cancers have clinical responses, including partial and complete responses in some patients;
We have shifted our development strategy for first approval from 1L NSCLC to 2L cervical cancer because of increasing hurdles and correspondingly longer timelines.
Advance our cervical cancer trial of AGEN1884 and AGEN2034 combination is currently enrolling patients.
File an IND on next generation CTLA-4 (AGEN1181) designed to improve T cell priming and Treg depletion;
File INDs for our bispecific antibodies designed to condition the tumor microenvironment through regulatory T cell depletion and other undisclosed mechanisms;
Advance efforts to launch a combination trial with CTLA-4, PD1 & our neoantigen vaccine, AutoSynVax + QS-21.
Manufacturing Update
Supplied GMP material for clinical programs and delivered commercial grade AGEN1884
We are also on track having already filled vials of commercial grade AGEN2034
QS-21 Stimulon update
SHINGRIX is the most effective shingles vaccines; GSK commercial sales projections have nearly tripled from expectations earlier in the year
AgenTus Cell Therapy Business
IND filing for lead candidate in 2019
First Quarter 2018 Financial Results

Cash and cash equivalents were $52.3 million and $60.2 million at March 31, 2018 and December 31, 2017 respectively.

For the first quarter ended March 31, 2018, we reported research and development expenses of $29.4 million, and $32.6 million for the same period in 2017. Our net loss of for the three months ended March 31, 2018 is $54.3 million or $0.53 per share compared to a net loss for same period in 2017 of $17.1 million, or $0.18 per share. The increased net loss reflects unfavorable items effecting the current quarter and favorable items effecting the same period last year; including, the loss on the extinguishment of our debt, increased change in the non-cash contingent considerations fair value adjustment as well as reduced revenue due to an accelerated milestone received during the first quarter of 2017 from Incyte.

Conference Call, Webcast and Prepared Statement Information

Agenus executives will host a conference call on Monday, May 7, 2018 at 11:00 a.m. Eastern Time. To access the live call, dial (844) 492-3727 (domestic) and (412) 317-5118 (international). Ask to be joined into the Agenus call. The call will also be webcast and will be accessible from the Company’s website at View Source or via the following link: View Source A replay will be available on the Company’s website approximately two hours after the call and will remain available for 90 days.

PROVECTUS BIOPHARMACEUTICALS COMPLETES ENROLLMENT OF PHASE 1B TRIAL OF PV-10 IN COMBINATION WITH KEYTRUDA® IN PATIENTS WITH METASTATIC MELANOMA

On May 7, 2018 Provectus reported the completion of enrollment of 24 patients with metastatic melanoma into the Phase 1b portion of the Company’s Phase 1b/2 study of intralesional ("IL") PV-10 in combination with KEYTRUDA (pembrolizumab), Merck & Co.’s systemic anti-PD-1 (programmed death receptor-1) antibody (ClinicalTrials.gov identifier: NCT02557321) (Press release, Provectus Pharmaceuticals, MAY 7, 2018, View Source [SID1234526176]). This study’s endpoints include those currently suitable for a registration trial.

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Provectus also plans to present comprehensive Phase 1b data in the first half of 2019.

Provectus Biopharmaceuticals, Inc. (OTCQB: PVCT, www.provectusbio.com) ("Provectus" or the "Company") is a clinical-stage biotechnology company developing PV-10 as the first small molecule oncolytic immunotherapy for solid tumor cancers.

About PV-10

Provectus’ lead investigational oncology drug product, PV-10, the first small molecule oncolytic immunotherapy, can induce immunogenic cell death. PV-10 is undergoing clinical study for adult solid tumor cancers, like melanoma and cancers of the liver, and preclinical study for pediatric cancers.

About our Phase 1b/2 Study of PV-10 + KEYTRUDA for Metastatic Melanoma

Patients with metastatic melanoma having at least one injectable cutaneous or soft tissue lesion were eligible for participation in the Phase 1b portion of the study and received the combination of IL PV-10 and KEYTRUDA every three weeks for up to five cycles (i.e., for up to 12 weeks, with no further PV-10 administered after week 12), followed by only KEYTRUDA every three weeks for up to 24 months. The primary endpoint for the Phase 1b trial is safety and tolerability. Objective response rate and progression-free survival are key secondary endpoints; both are assessed via RECIST 1.1 after five treatment cycles, and then every 12 weeks thereafter.

Allergan to Present at the Bank of America Merrill Lynch Healthcare Conference

On May 7, 2018 Allergan plc (NYSE: AGN), a leading global biopharmaceutical company, reported that Chief Commercial Officer William Meury will present at the Bank of America Merrill Lynch Healthcare Conference in Las Vegas, Nevada (Press release, Allergan, MAY 7, 2018, View Source(1) [SID1234526160]). The presentation will begin at 8:00 a.m. Pacific Time (11:00 a.m. Eastern Time) on Thursday, May 17, 2018.

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The presentation will be webcast live and can be accessed on Allergan’s Investor Relations website at www.allergan.com/investors. The webcast can also be accessed through the following URL: https://www.veracast.com/webca…

An archived version will be available within approximately one hour of the live presentation, and can be accessed at the same location for 90 days.

FDA Grants Priority Review to Genentech’s Cancer Immunotherapy Tecentriq (Atezolizumab) for Initial Treatment of People With a Specific Type of Metastatic Lung Cancer

On May 6, 2018 Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), reported that the U.S. Food and Drug Administration (FDA) has accepted the company’s supplemental Biologics License Application (sBLA) and granted Priority Review for TECENTRIQ (atezolizumab), in combination with Avastin (bevacizumab), paclitaxel and carboplatin (chemotherapy), for the initial (first-line) treatment of people with metastatic non-squamous non-small cell lung cancer (NSCLC) (Press release, Genentech, MAY 6, 2018, View Source [SID1234526164]). The FDA is expected to make a decision on approval by September 5, 2018. A Priority Review designation is granted to medicines that the FDA has determined to have the potential to provide significant improvements in the treatment, prevention or diagnosis of a disease.

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"Our Phase III results showed TECENTRIQ in combination with Avastin, paclitaxel and carboplatin has the potential to provide a significant survival benefit in the initial treatment of metastatic non-squamous non-small cell lung cancer," said Sandra Horning, M.D., chief medical officer and head of Global Product Development. "We are working closely with the FDA to bring this treatment regimen to people with this type of lung cancer as soon as possible."

This sBLA is based on results from the Phase III IMpower150 study, which met its co-primary endpoints of overall survival (OS) and progression-free survival (PFS) in the initial treatment of people with advanced non-squamous NSCLC. The safety profile of the combination was consistent with the safety profiles of the individual medicines, and no new safety signals were identified.

TECENTRIQ is currently approved by the FDA to treat people with metastatic NSCLC who have disease progression during or following platinum-containing chemotherapy, and have progressed on an appropriate FDA-approved targeted therapy if their tumor has ALK or EGFR gene abnormalities.

About the IMpower150 study

IMpower150 is a multicenter, open-label, randomized, controlled Phase III study evaluating the efficacy and safety of TECENTRIQ in combination with carboplatin and paclitaxel with or without Avastin in people with stage IV non-squamous NSCLC who had not been treated with chemotherapy for their advanced disease. It enrolled 1,202 people, of which those with ALK and EGFR mutations were excluded from the primary intention-to-treat (ITT) analysis. People were randomized (1:1:1) to receive:

TECENTRIQ plus carboplatin and paclitaxel (Arm A), or
TECENTRIQ and Avastin plus carboplatin and paclitaxel (Arm B), or
Avastin plus carboplatin and paclitaxel (Arm C, control arm).
The co-primary endpoints were OS and PFS, as determined by the investigator using Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST v1.1). The primary analysis of the co-primary PFS endpoint in IMpower150 was assessed in two populations: all randomized people without an ALK or EGFR genetic mutation (intention-to-treat wild-type) and in a subgroup of people who had a specific biomarker (T-effector "Teff" gene signature expression). The co-primary OS endpoint was assessed in all randomized people without an ALK or EGFR genetic mutation (intention-to-treat wild-type). Key secondary endpoints included investigator-assessed PFS and OS, safety in the ITT population and in EGFR and ALK mutation subgroups.

About lung cancer

According to the American Cancer Society, it is estimated that more than 234,000 Americans will be diagnosed with lung cancer in 2018, and NSCLC accounts for 85 percent of all lung cancers. It is estimated that approximately 60 percent of lung cancer diagnoses in the United States are made when the disease is in the advanced stages.

About the TECENTRIQ (atezolizumab) and Avastin (bevacizumab) combination

There is a strong scientific rationale to support further investigation of TECENTRIQ plus Avastin in combination. We are investigating this combination in a broad range of cancers, including first-line advanced NSCLC. Avastin, in addition to its anti-angiogenic effects, may further enhance TECENTRIQ’s ability to restore anti-cancer immunity by inhibiting VEGF-related immunosuppression, promoting T-cell tumor infiltration and enabling priming and activation of T-cell responses against tumor antigens.

About TECENTRIQ (atezolizumab)

TECENTRIQ is a monoclonal antibody designed to bind with a protein called PD-L1. TECENTRIQ is designed to bind to PD-L1 expressed on tumor cells and tumor-infiltrating immune cells, blocking its interactions with both PD-1 and B7.1 receptors. By inhibiting PD-L1, TECENTRIQ may enable the re-activation of T cells. TECENTRIQ may also affect normal cells.

About Avastin (bevacizumab)

Avastin is a prescription-only medicine that is a solution for intravenous infusion. It is a biologic antibody designed to specifically bind to a protein called vascular endothelial growth factor (VEGF) that plays an important role throughout the lifecycle of the tumor to develop and maintain blood vessels, a process known as angiogenesis. Avastin is designed to interfere with the tumor blood supply by directly binding to the VEGF protein to prevent interactions with receptors on blood vessel cells. The tumor blood supply is thought to be critical to a tumor’s ability to grow and spread in the body (metastasize).

TECENTRIQ U.S. Indication (pronounced ‘tē-SEN-trik’)

TECENTRIQ is a prescription medicine used to treat:

a type of bladder and urinary tract cancer called urothelial carcinoma.

TECENTRIQ may be used when your bladder cancer:
has spread or cannot be removed by surgery (advanced urothelial carcinoma), and
you are not able to take chemotherapy that contains a medicine called cisplatin, or
you have tried chemotherapy that contains platinum, and it did not work or is no longer working
The approval of TECENTRIQ in these patients is based on a study that measured response rate and duration of response. There is an ongoing study to confirm clinical benefit.

A type of lung cancer called non-small cell lung cancer (NSCLC).

TECENTRIQ may be used when your lung cancer:
has spread or grown, and
you have tried chemotherapy that contains platinum, and it did not work or is no longer working
If your tumor has an abnormal EGFR or ALK gene, you should have also tried an FDA-approved therapy for tumors with these abnormal genes, and it did not work or is no longer working.

It is not known if TECENTRIQ is safe and effective in children.

Important Safety Information

Important Information About TECENTRIQ

TECENTRIQ can cause the immune system to attack normal organs and tissues in many areas of the body and can affect the way they work. These problems can sometimes become serious or life threatening and can lead to death.

Patients should call or see their healthcare provider right away if they get any symptoms of the following problems or these symptoms get worse.

TECENTRIQ can cause serious side effects, including:

Lung problems (pneumonitis ) – signs and symptoms may include new or worsening cough, shortness of breath, and chest pain
Liver problems (hepatitis) – signs and symptoms of hepatitis may include yellowing of the skin or the whites of the eyes, severe nausea or vomiting, pain on the right side of the stomach area (abdomen), drowsiness, dark urine (tea colored), bleeding or bruising more easily than normal, and feeling less hungry than usual
Intestinal problems (colitis) – signs and symptoms of colitis may include diarrhea (loose stools) or more bowel movements than usual, blood or mucous in the stools or dark, tarry, sticky stools, and severe stomach area (abdomen) pain or tenderness
Hormone gland problems (especially the thyroid, adrenal glands, pancreas, and pituitary) – signs and symptoms that the hormone glands are not working properly may include headaches that will not go away or unusual headaches, extreme tiredness, weight gain or weight loss, dizziness or fainting, feeling more hungry or thirsty than usual, hair loss, changes in mood or behavior (such as decreased sex drive, irritability, or forgetfulness), feeling cold, constipation, voice gets deeper, urinating more often than usual, nausea or vomiting, and stomach area (abdomen) pain
Problems in other organs – signs and symptoms may include severe muscle weakness, numbness or tingling in hands or feet, confusion, blurry vision, double vision, or other vision problems, changes in mood or behavior, extreme sensitivity to light, neck stiffness, eye pain or redness, skin blisters or peeling, chest pain, irregular heartbeat, shortness of breath, or swelling of the ankles
Severe infections – signs and symptoms of infection may include fever, cough, flu-like symptoms, pain when urinating, and frequent urination or back pain
Severe infusion reactions – signs and symptoms of infusion reactions may include chills or shaking, itching or rash, flushing, shortness of breath or wheezing, swelling of the face or lips, dizziness, fever, feeling like passing out, and back or neck pain
Getting medical treatment right away may help keep these problems from becoming more serious. A healthcare provider may treat a patient with corticosteroid or hormone replacement medicines. A healthcare provider may delay or completely stop treatment with TECENTRIQ if a patient has severe side effects.

Before receiving TECENTRIQ, patients should tell their healthcare provider about all of their medical conditions, including if they:

Have immune system problems (such as Crohn’s disease, ulcerative colitis, or lupus); have had an organ transplant; have lung or breathing problems; have liver problems; have a condition that affects their nervous system (such as myasthenia gravis or Guillain-Barre syndrome); or are being treated for an infection
Are pregnant or plan to become pregnant. TECENTRIQ can harm an unborn baby. If patients are able to become pregnant, they should use an effective method of birth control during treatment and for at least 5 months after the last dose of TECENTRIQ
Are breastfeeding or plan to breastfeed. It is not known if TECENTRIQ passes into the breast milk. Do not breastfeed during treatment and for at least 5 months after the last dose of TECENTRIQ
Patients should tell their healthcare provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

The most common side effects of TECENTRIQ in people with urothelial carcinoma include:

feeling tired
decreased appetite
nausea
constipation
urinary tract infection
diarrhea
fever
The most common side effects of TECENTRIQ in people with non-small cell lung cancer include:

feeling tired
decreased appetite
muscle pain
cough
shortness of breath
TECENTRIQ may cause fertility problems in females, which may affect the ability to have children. Patients should talk to their healthcare provider if they have concerns about fertility.

These are not all the possible side effects of TECENTRIQ. Patients should ask their healthcare provider or pharmacist for more information. Patients should call their doctor for medical advice about side effects.

Report side effects to the FDA at 1-800-FDA-1088 or View Source Report side effect to Genentech at 1-888-835-2555.

Please visit View Source for the TECENTRIQ full Prescribing Information for additional Important Safety Information.

Avastin Indications:

Metastatic colorectal cancer (mCRC) for first- or second-line treatment in combination with intravenous 5-fluorouracil–based chemotherapy. It is also approved to treat mCRC for second-line treatment when used with fluoropyrimidine-based (combined with irinotecan or oxaliplatin) chemotherapy after cancer progresses following a first-line treatment that includes Avastin.
Avastin is not approved for use after the primary treatment of colon cancer that has not spread to other parts of the body.
Advanced nonsquamous non–small cell lung cancer (NSCLC) in combination with carboplatin and paclitaxel in people who have not received chemotherapy for their advanced disease.
Metastatic kidney cancer (mRCC) when used with interferon alfa.
Glioblastoma (GBM) in adult patients whose cancer has progressed after prior treatment (recurrent or rGBM).
Advanced cervical cancer (CC) in combination with paclitaxel and cisplatin or paclitaxel and topotecan is approved to treat persistent, recurrent, or metastatic cancer of the cervix.
Recurrent ovarian cancer (rOC) . Avastin in combination with paclitaxel, pegylated liposomal doxorubicin or topotecan, is approved to treat platinum-resistant recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer (prOC) in women who received no more than two prior chemotherapy treatments. Avastin, either in combination with carboplatin and paclitaxel or with carboplatin and gemcitabine, followed by Avastin alone, is approved for the treatment of patients with platinum-sensitive recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer (psOC).
Possible serious side effects

Everyone reacts differently to Avastin therapy. So it’s important to know what the side effects are. Although some people may have a life-threatening side effect, most do not. Their doctor will stop treatment if any serious side effects occur. Patients should talk to their doctor if there are any signs of these side effects.

Most serious side effects (not common, but sometimes fatal):

GI perforation. A hole that develops in the stomach or intestine. Symptoms include pain in the abdomen, nausea, vomiting, constipation, or fever
Wounds that don’t heal. A cut made during surgery can be slow to heal or may not fully heal. Avastin should not be used for at least 28 days before or after surgery and until surgicalwounds are fully healed
Serious bleeding. This includes vomiting or coughing up blood; bleeding in the stomach, brain, or spinal cord; nosebleeds; and vaginal bleeding. If a patient has recently coughed up blood or had serious bleeding, they should be sure to tell their doctor
Other possible serious side effects

Abnormal passage in the body. This type of passage—known as a fistula—is an irregular connection from one part of the body to another and can sometimes be fatal
Severe high blood pressure. Blood pressure that severely spikes or shows signs of affecting the brain. Blood pressure should be monitored every 2 to 3 weeks while on Avastin and after stopping treatment
Kidney problems. These may be caused by too much protein in the urine and can sometimes be fatal
Infusion reactions. These were uncommon with the first dose (less than 3% of patients). 0.2% of patients had severe reactions. Infusion reactions include high blood pressure or severe high blood pressure that may lead to stroke, trouble breathing, decreased oxygen in red blood cells, a serious allergic reaction, chest pain, headache, tremors, and excessive sweating. The patient’s doctor or nurse will monitor for signs of infusion reactions
Severe stroke or heart problems. These may include blood clots, mini-stroke, heart attack, chest pain, and the heart may become too weak to pump blood to other parts of the body (congestive heart failure). These can sometimes be fatal
Nervous system and vision problems. Signs include headache, seizure, high blood pressure, sluggishness, confusion, and blindness
Side effects seen most often

In clinical studies across different types of cancer, some patients experienced the following side effects:

High blood pressure
Too much protein in the urine
Nosebleeds
Rectal bleeding
Back pain
Headache
Taste change
Dry skin
Inflammation of the skin
Inflammation of the nose
Watery eyes
Avastin is not for everyone

Patients should talk to their doctor if they are:

Undergoing surgery. Avastin should not be used for 28 days before or after surgery and until surgical wounds are fully healed
Pregnant or think they are pregnant. Data have shown that Avastin may harm a woman’s unborn baby. Birth control should be used while patients are on Avastin. If Avastin is stopped, patients should keep using birth control for 6 months before trying to become pregnant
Planning to become pregnant. Taking Avastin could cause a woman’s ovaries to stop working and may impair her ability to have children
Breastfeeding. Breastfeeding while on Avastin may harm the baby and is therefore not recommended
Patients should talk with their doctor if they have any questions about their condition or treatment.

Report side effects to the FDA at (800) FDA-1088 or View Source Report side effects to Genentech at (888) 835-2555.

For full Prescribing Information on Avastin please visit View Source

About Genentech in Personalized Cancer Immunotherapy

For more than 30 years, Genentech has been developing medicines with the goal to redefine treatment in oncology. Today, we’re investing more than ever to bring personalized cancer immunotherapy (PCI) to people with cancer. The goal of PCI is to provide each person with a treatment tailored to harness his or her own immune system to fight cancer. Genentech is studying more than 20 investigational medicines, 10 of which are in clinical trials. In every study we are evaluating biomarkers to identify which people may be appropriate candidates for our medicines. For more information visit View Source

About Genentech in Lung Cancer

Lung cancer is a major area of focus and investment for Genentech, and we are committed to developing new approaches, medicines and tests that can help people with this deadly disease. Our goal is to provide an effective treatment option for every person diagnosed with lung cancer. We currently have four approved medicines to treat certain kinds of lung cancer and more than 10 medicines being developed to target the most common genetic drivers of lung cancer or to boost the immune system to combat the disease.

Agios Provides Business Update on Discovery Research Strategy and Pipeline, Progress on Clinical Programs, Commercial Launch Preparations and Reports First Quarter 2018 Financial Results at Investor Day

On May 4, 2018 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, is hosting an Investor Day in New York City today. During the event, reported that the company will provide a comprehensive business update and report financial results for the first quarter ended March 31, 2018 (Press release, Agios Pharmaceuticals, MAY 4, 2018, View Source [SID1234526111]). The presentations will highlight how Agios’ drug discovery platform and broad clinical portfolio set Agios on the path to become a sustainable, multi-product biopharmaceutical company. The event will be webcast today starting at 8:00 a.m. ET at investor.agios.com.

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"As we prepare to launch our second Agios-discovered and first wholly owned medicine later this year, we continue to invest in our productive drug discovery engine and advance a robust pipeline of first-in-class medicines," said David Schenkein, M.D., chief executive officer at Agios. "Our first quarter progress against that objective was highlighted by the NDA acceptance of TIBSOVO in IDH1m relapsed or refractory AML and multiple clinical trial initiations, including dosing the first patient with our MAT2A inhibitor AG-270 and the start of our AG-348 pivotal program in PK deficiency."

HIGHLIGHTS FROM INVESTOR DAY PRESENTATIONS

• Communicated a robust research pipeline consisting of nine advanced drug discovery programs against novel targets across oncology, rare genetic diseases and metabolic immuno-oncology with the potential to deliver multiple INDs over the next 24 months.

• Expanded rare genetic disease portfolio:

• The company disclosed active research programs in three rare genetic diseases: phenylketonuria (PKU), erythroid porphyria and Friedreich’s ataxia
LOGO

• The most advanced research program is in PKU, where Agios has developed a novel approach to stabilize the mutant phenylalanine hydroxylase (PAH) protein and has demonstrated significantly decreased blood phenylalanine levels in a severe pre-clinical model of the disease. PKU is an autosomal recessive disease caused by mutations in the PAH gene affecting approximately 16,000 patients in the U.S.1

• Updated clinical milestones to advance the development of isocitrate dehydrogenase (IDH) 1 inhibitors in solid tumors:

• Glioma pivotal development strategy expected to be finalized by year-end 2018

• Completion of enrollment of ClarIDHy, a global, registration-enabling randomized Phase 3 study for ivosidenib in IDH1m positive advanced cholangiocarcinoma, accelerated to the first half of 2019

• Announced acceptance of the following presentations at 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting:

• Updated data from the expansion phase of the ongoing Phase 1 study of ivosidenib in IDH1m relapsed or refractory (R/R) acute myeloid leukemia (AML)

• Updated data from the ongoing Phase 1/2 combination trial of enasidenib or ivosidenib with VIDAZA in patients with newly diagnosed AML with an IDH2 or IDH1 mutation ineligible for intensive chemotherapy

• First clinical data from the Phase 1 study of AG-881 in advanced IDHm positive solid tumors, including glioma

• Completed commercial infrastructure build, including the deployment of an expanded sales force, to successfully launch TIBSOVO (ivosidenib) within 48 hours of potential FDA approval.
FIRST QUARTER 2018 HIGHLIGHTS & RECENT PROGRESS

• Initiated ACTIVATE-T, a single-arm pivotal trial for AG-348, in adult pyruvate kinase (PK) deficiency patients who receive regular blood transfusions.

• Initiated PEAK, a global registry, for adult and pediatric patients with PK deficiency.

• Initiated a perioperative ‘window’ trial with ivosidenib and AG-881 in IDHm low-grade glioma to further investigate their effects on brain tumor tissue.

• Initiated a Phase 1 dose-escalation trial for AG-270, a first-in-class methionine adenosyltransferase 2a (MAT2A) inhibitor, in patients with methylthioadenosine phosphorylase (MTAP)-deleted tumors.

• Announced FDA acceptance, priority review and a Prescription Drug User Fee Act (PDUFA) action date of August 21, 2018 for the new drug application (NDA) for TIBSOVO (ivosidenib) for the treatment of patients with R/R AML with an IDH1 mutation.

• Completed an underwritten public offering of 8,152,986 shares of common stock at the offering price of $67.00 per share, resulting in proceeds to the company, net of underwriting discounts and commissions, of approximately $516.2 million.
LOGO

UPCOMING 2018 MILESTONES & EXPECTED DATA PRESENTATIONS

The company expects to achieve the following additional milestones in 2018:

Cancer:

Potential approval and commercialization of TIBSOVO (ivosidenib) in the United States for R/R AML with an IDH1 mutation in the third quarter of 2018.

Submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for TIBSOVO (ivosidenib) for the treatment of patients with R/R AML and an IDH1 mutation in the fourth quarter of 2018.

Support, in collaboration with Celgene, the initiation of HO150, an intergroup sponsored, global, registration-enabling Phase 3 trial combining ivosidenib or enasidenib with standard induction and consolidation chemotherapy in frontline AML patients with an IDH1 or IDH2 mutation in the fourth quarter of 2018.

Present updated data from the ongoing Phase 1 combination trial of enasidenib or ivosidenib with standard-of-care intensive chemotherapy in patients with newly diagnosed AML with an IDH2 or IDH1 mutation to the 2018 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition.
Rare Genetic Diseases:

Initiate ACTIVATE, a global, placebo-controlled, pivotal trial for AG-348 in approximately 80 adults with PK deficiency who do not receive regular blood transfusions in the second quarter of 2018.

Initiate a Phase 2 proof of concept trial of AG-348 in thalassemia in the fourth quarter of 2018.
Research:

Submit an investigational new drug (IND) application for our newest development candidate, AG-636, an inhibitor of the metabolic enzyme dihydroorotate dehydrogenase (DHODH) for the treatment of hematologic malignancies in the fourth quarter of 2018.
FIRST QUARTER 2018 FINANCIAL RESULTS & CASH GUIDANCE

Revenue for the quarter ended March 31, 2018 was $8.8 million, which includes $7.4 million of collaboration revenue and $1.4 million of royalty revenue from net sales of IDHIFA. Revenue for the quarter ended March 31, 2017 was $10.5 million and consisted solely of collaboration revenue. The decrease in collaboration revenue recognized for the quarter ended March 31, 2018 compared to the comparable period in 2017 was primarily driven by adoption of the new revenue recognition standard.

Research and development (R&D) expenses were $78.2 million, including $8.6 million of stock-based compensation expense, for the quarter ended March 31, 2018, compared to $62.7 million, including $7.0 million in stock-based compensation expense, for the comparable period in 2017. The increase in R&D expense was primarily attributable to start-up costs for the AG-348 pivotal program in PK deficiency, including the initiation of the ACTIVATE-T trial. R&D expense also increased as a result of the initiation of a Phase 1 dose-escalation study of AG-270, our first-in-class MAT2A inhibitor, and IND enabling activities for AG-636, our DHODH inhibitor.

General and administrative (G&A) expenses were $24.6 million, including $5.9 million of stock-based compensation expense, for the quarter ended March 31, 2018, compared to $14.8 million, including $3.7 million of stock-based compensation expense, for the quarter ended March 31, 2017. The increase in G&A expense was primarily attributable to the growth in our U.S. commercial organization in order to support the expected launch of TIBSOVO (ivosidenib) in the third quarter of 2018.

Net loss for the quarter ended March 31, 2018 was $90.8 million, compared to a net loss of $66.2 million for the quarter ended March 31, 2017.

Cash, cash equivalents and marketable securities as of March 31, 2018 were $994.7 million, compared to $567.8 million as of December 31, 2017. The increase in cash was driven by the net proceeds of $516.2 million from the January follow on offering, $4.4 million of cost reimbursements under our collaboration agreements with Celgene and $12.3 million received from employee stock transactions. This was offset by expenditures to fund operations of $104.8 million during the quarter ended March 31, 2018.

The company expects that its cash, cash equivalents and marketable securities as of March 31, 2018, together with the anticipated product and royalty revenue, anticipated interest income, and anticipated expense reimbursements, but excluding any additional program-specific milestone payments, will enable the company to fund its anticipated operating expenses and capital expenditure requirements through at least the end of 2020.

WEBCAST INFORMATION

The live webcast from today’s event can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. The archived webcast will be available on the company’s website after the event.